On August 14, 2025 CARsgen Therapeutics Holdings Limited (Stock Code: 2171.HK), a company focused on developing innovative CAR T-cell therapies, reported its 2025 Interim Results (Press release, Carsgen Therapeutics, AUG 14, 2025, View Source [SID1234655321]).

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Business Highlights

Cash and bank balances were around RMB1,261 million as of June 30, 2025. Cash and cash equivalents and deposits at the end of 2025 are expected to be not less than RMB1,100 million. We expect to have adequate cash into the 2028 excluding subsequent cash inflows.
During H1 2025, certification and regulatory filings for zevor-cel have been completed in more than 20 provinces or cities. CARsgen has received a total of 111 confirmed orders from its commercialization partner Huadong Medicine.
The Center for Drug Evaluation (CDE) of the National Medical Products Administration (NMPA) of China has accepted the New Drug Application (NDA) for satri-cel.
The results of satri-cel confirmatory Phase II trial in China have been published in The Lancet and at the 2025 ASCO (Free ASCO Whitepaper) Annual Meeting.
Multiple allogeneic CAR-T products are in development, covering treatment areas such as hematologic malignancies, solid tumors, and autoimmune diseases.
CARsgen introduced Zhuhai SB Xinchuang to accelerate allogeneic CAR-T development in mainland China.
Dr. Zonghai Li, Founder, Chairman of the Board, Chief Executive Officer, and Chief Scientific Officer of CARsgen Therapeutics, said, "In the first half of 2025, we made significant strides across technology innovation, product development, and commercialization. Zevor-cel sales surged, while satri-cel became the world’s first CAR-T targeting solid tumors to file an NDA. We are also advancing multiple allogeneic CAR-T therapies to enhance clinical outcomes and expand patient access."

Financial Highlights

CARsgen’s revenue was around RMB51 million for the six months ended June 30, 2025, mainly from zevor-cel, which was calculated on the basis of ex-works price, rather than end-of-market prices. Our revenue is recognized upon completion of ex-works delivery of products. Due to the inherent time cycle of CAR-T manufacturing, there is a discrepancy between the number of orders obtained from Huadong Medicine and number of ex-works deliveries. CARsgen’s gross profit was around RMB29 million for the six months ended June 30, 2025. In the commercialization stage, we are demonstrating a strong cost competitive advantage, which is mainly due to self-manufacture for plasmids and vectors with stable output and high yield per batch.

Cash and bank balances were around RMB1,261 million as of June 30, 2025, representing a decrease of around RMB218 million from around RMB1,479 million as of December 31, 2024. The decrease was mainly due to the payment of research and development expenses, administrative expenses and investment of capital expenditure. Cash and cash equivalents and deposits at the end of 2025 are expected to be not less than RMB1,100 million. We expect to have adequate cash into the 2028 excluding subsequent cash inflows.

Zevor-cel Demonstrates Rapid Sales Growth

Zevorcabtagene autoleucel (zevor-cel, R&D code: CT053) is an autologous fully human CAR T-cell product against B-cell maturation antigen (BCMA) approved by the NMPA of China for the treatment of adult patients with relapsed or refractory multiple myeloma (R/R MM) who have progressed after at least 3 prior lines of therapy (including a proteasome inhibitor and an immunomodulatory agent).

CARsgen entered into a collaboration agreement with Huadong Medicine (000963.SZ) for the commercialization of zevor-cel in mainland China. In terms of commercialization, Huadong Medicine has established a dedicated, professional, and comprehensive commercial team to promote the use of zevor-cel and has been utilizing China’s multi-layered insurance system to improve patient accessibility. During the first half of 2025, certification and regulatory filings for zevor-cel have been completed in more than 20 provinces or cities and we have received a total of 111 confirmed orders from Huadong Medicine. We anticipate that growth of sales revenue of zevor-cel will further accelerate with continuous marketing activities and broader insurance coverage.

Satri-cel NDA Accepted for Review in China

Satricabtagene autoleucel (satri-cel, R&D code: CT041) is an autologous humanized CAR T-cell product against Claudin18.2 (CLDN18.2). In June 2025, the CDE of NMPA of China has accepted the New Drug Application (NDA) for satri-cel for the treatment of Claudin18.2-positive advanced gastric/gastroesophageal junction adenocarcinoma (G/GEJA) in patients who have failed at least two prior lines of therapy. Satri-cel is the first and only CAR T-cell product globally for which an NDA submitted for the treatment of solid tumors. Satri-cel was granted Priority Review in May 2025 and Breakthrough Therapy Designation (BTD) in March 2025 by the CDE.

The results of satri-cel confirmatory Phase II trial (NCT04581473) in China have been published in The Lancet and were orally presented at the 2025 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting. Satri-cel demonstrated significant progression-free survival (PFS) improvement and a clinically meaningful overall survival (OS) benefit with a manageable safety profile, compared to standard therapy.

Multiple Allogeneic CAR-T Product Candidates in Development

CARsgen has been advancing differentiated allogeneic CAR T-cell products utilizing the proprietary THANK-uCAR platform. CARsgen has recently developed the THANK-u PlusTM platform as an enhanced version of THANK-uCAR to address the potential impact of NKG2A expression levels on therapeutic efficacy of the allogeneic CAR T-cells.

CT0596 is an allogeneic BCMA-targeted CAR T-cell product utilizing THANK-u Plus platform for the treatment of R/R MM and R/R plasma cell leukemia (PCL). The investigator-initiated trials (IITs) are ongoing in China. Preliminary clinical data for CT0596 were released in May 2025 on CARsgen’s official website. Based on the preliminary safety and efficacy data, CT0596 demonstrated favorable tolerability and encouraging efficacy signals in R/R MM patients across all predefined dose levels, with CAR-T expansion observed.

In addition, there are several allogeneic CAR T-cell products under development:

KJ-C2219: Targeting CD19/CD20, for the treatment of hematologic malignancies and autoimmune diseases. An IIT for relapsed/refractory B-cell non-Hodgkin lymphoma (R/R B-NHL) has been initiated at the end of 2024. A separate IIT for systemic lupus erythematosus (SLE) and systemic sclerosis (SSc) has been initiated in the first half of 2025.
KJ-C2320: Targeting CD38, for the treatment of acute myeloid leukemia (AML). An IIT has been initiated at the end of 2024.
KJ-C2114: For the treatment of solid tumors.
KJ-C2526: Targeting NKG2DL, for the treatment of AML, other malignancies, and senescence.
On February 25, 2025, CARsgen has entered into the agreements (the "Agreements") with an investment fund (the "Investor") managed by Zhuhai Hengqin SB Xinchuang Equity Investment Management Enterprise (Limited Partnership), pursuant to which, among others, the Investor has agreed to subscribe to additional registered capital of UCARsgen Biotech Limited ("UCARsgen") at a cash consideration of RMB80,000,000, representing 8% stake of the enlarged registered capital of UCARsgen. Upon the completion of the capital increase, CARsgen’s share in UCARsgen will be diluted from 100% to 92%.

UCARsgen is a China-based new drug discovery biotechnology company focused on allogeneic CAR T-cell therapies for the treatment of hematologic malignancies. Under the Agreements, UCARsgen has secured the exclusive rights in mainland China for the research, development, manufacture, and commercialization of the following allogeneic CAR T-cell products from CARsgen: the BCMA-targeted allogeneic CAR T-cell therapy for the treatment of multiple myeloma and plasma cell leukemia and the CD19/CD20 dual-targeted allogeneic CAR T-cell therapy for the treatment of B-cell malignancies (excluding indications for the treatment of autoimmune diseases).

On August 14, 2025 IO Biotech (Nasdaq: IOBT), a clinical-stage biopharmaceutical company developing novel, immune-modulatory, off-the-shelf therapeutic cancer vaccines, reported financial results and business highlights for the second quarter of 2025 (Press release, IO Biotech, AUG 14, 2025, View Source [SID1234655304]).

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"In the second quarter, we achieved a significant milestone advancing our pipeline of novel cancer therapies with the readout of the topline results of the Phase 3 trial for Cylembio, our potentially first-in-class, immune-modulatory, off-the-shelf therapeutic cancer vaccine, being investigated in advanced melanoma," said Mai-Britt Zocca, PhD, President and CEO of IO Biotech. "With the notable safety profile and clinical improvement observed in this trial, we are focused on discussing the results with the FDA and determining the next steps for a potential submission of a Biologics License Application (BLA) for the treatment of advanced melanoma."

Recent Business Highlights

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The company announced topline results of its Phase 3 pivotal trial (IOB-013/KN-D18) evaluating the company’s lead investigational vaccine, Cylembio (imsapepimut and etimupepimut, adjuvanted), in combination with Merck’s (known as MSD outside of the United States and Canada) anti-PD-1 therapy KEYTRUDA (pembrolizumab) for the treatment of advanced melanoma. The trial evaluated Cylembio in combination with pembrolizumab vs. pembrolizumab alone as a first-line treatment in 407 patients with unresectable or metastatic (advanced) melanoma. In the study, Cylembio plus pembrolizumab demonstrated clinical improvement in progression free survival (PFS) compared to pembrolizumab alone, but statistical significance was narrowly missed on the PFS primary endpoint. Based on these results, IO Biotech plans to meet with the United States (US) Food and Drug Administration (FDA) this fall to discuss the totality of data and determine next steps for a potential submission of a Biologics License Application (BLA) for the treatment of advanced melanoma.

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The company continues to expect initial data from the perioperative Phase 2 solid tumor basket trial (IOB-032/PN-E40), studying treatment with Cylembio in combination with pembrolizumab in patients with resectable squamous cell carcinoma of the head and neck (SCCHN) and melanoma that completed enrollment in January, as well as longer-term data from the company’s other ongoing Phase 2 basket trial, IOB-022/KN-D38, in patients with advanced SCCHN or non-small cell lung cancer (NSCLC), to be available in the second half of 2025 and presented at a medical meeting in 2026.

Upcoming Investor Conferences Participation

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Morgan Stanley 23rd Annual Global Healthcare Conference: Mai-Britt Zocca, PhD, President and CEO, Amy Sullivan, CFO, and Qasim Ahmad, MD, CMO, will participate in a fireside chat beginning at 7:45 AM ET on September 9, 2025.

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H.C. Wainwright 27th Annual Global Investment Conference: Mai-Britt Zocca, PhD, President and CEO, will give a company presentation beginning at 8:00 AM ET on September 10, 2025.

The webcasts for these two upcoming conferences will be available from the Investors section of the company’s website at View Source

Second Quarter 2025 Financial Results

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Net loss for the three months ended June 30, 2025, was $26.2 million, compared to $20.7 million for the three months ended June 30, 2024.

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Research and development expenses were $16.7 million for the three months ended June 30, 2025, compared to $15.8 million for the three months ended June 30, 2024. The company recognized $0.6 million in research and development equity-based compensation for the three months ended June 30, 2025, compared to $0.7 million for the three months ended June 30, 2024.

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General and administrative expenses were $6.5 million for the three months ended June 30, 2025, compared to $5.7 million for the three months ended June 30, 2024. The company recognized $1.0 million in general and administrative equity-based compensation for the three months ended June 30, 2025 and 2024, respectively.

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Cash and cash equivalents as of June 30, 2025 were $28.1 million, compared to $60.0 million at December 31, 2024. During the three months ended June 30, 2025, the company used cash, cash equivalents and restricted cash of $9.0 million. The company has drawn down on the Tranche A and Tranche B loan facility in principal amount of €10.0 million and €12.5 million, respectively, on May 6, 2025 and July 4, 2025, each before payment of certain fees and transaction related expense. With the funds received from the second tranche of the EIB loan facility on July 4th, the company expects that it will have sufficient cash to run the company into the first quarter of 2026.

About Cylembio

Cylembio (imsapepimut and etimupepimut, adjuvanted) is an investigational, immune-modulatory, off-the-shelf therapeutic cancer vaccine candidate designed to kill both tumor cells and immune-suppressive cells in the tumor microenvironment (TME) by stimulating activation and expansion of T cells against indoleamine 2,3-dioxygenase 1 (IDO1) positive and/or programmed death-ligand 1 (PD-L1) positive cells. The company is currently conducting a pivotal Phase 3 trial (IOB-013/KN-D18; NCT05155254) investigating Cylembio in combination with Merck’s anti-PD-1 therapy, KEYTRUDA (pembrolizumab) versus pembrolizumab alone in patients with advanced melanoma, a Phase 2 basket trial (IOB-022/KN-D38; NCT05077709) investigating Cylembio in combination with pembrolizumab as first line treatment in patients with advanced solid tumors, and a Phase 2 basket trial (IOB-032/PN-E40; NCT05280314) investigating Cylembio in combination with pembrolizumab as neo-adjuvant/adjuvant treatment of patients with solid tumors. Enrollment in the Phase 3 trial was completed rapidly by December 2023 with topline results from this trial reported in the third quarter of 2025. Enrollment in the two ongoing company-sponsored Phase 2 clinical trials is now complete.

The clinical trials are sponsored by IO Biotech and conducted in collaboration with Merck, which is supplying pembrolizumab. IO Biotech maintains global commercial rights to Cylembio.

Cylembio is a registered trademark of IO Biotech ApS, a subsidiary of IO Biotech.

KEYTRUDA is a registered trademark of Merck Sharp & Dohme LLC, a subsidiary of Merck & Co., Inc., Rahway, NJ, USA (known as MSD outside of the US and Canada).

About the IOB-013/KN-D18 Pivotal Phase 3 Clinical Trial

IOB-013/KN-D18 (ClinicalTrials.gov: NCT05155254) is an open label, randomized Phase 3 pivotal clinical trial evaluating Cylembio in combination with Merck’s anti-PD-1 therapy, KEYTRUDA (pembrolizumab) versus pembrolizumab alone in patients with previously untreated, unresectable or metastatic (advanced) melanoma. Enrollment in the trial was completed by December 2023 with a total of 407 patients enrolled from more than 100 centers across the United States, Europe, Australia, Turkey, Israel and South Africa. The primary endpoint of the study was progression free survival. Secondary endpoints include overall response rate, overall survival, durable objective response rate, complete response rate, duration of response, time to complete response, disease control rate, and incidence of adverse events and serious adverse events (safety and tolerability). Biomarkers in the blood and tumor tissue will also be assessed as exploratory endpoints. The company reported topline results from this trial in the third quarter of 2025. IO Biotech is sponsoring the Phase 3 trial and Merck is supplying pembrolizumab.

About IOB-022/KN-D38 Phase 2 Solid Tumor Basket Trial

IOB-022/KN-D38 (NCT05077709) is a non-comparative, open label trial to investigate the safety and efficacy of Cylembio in combination with Merck’s anti-PD-1 therapy, KEYTRUDA (pembrolizumab) in the first-line treatment of metastatic non-small cell lung cancer (NSCLC) or metastatic squamous cell carcinoma of the head and neck (SCCHN) at sites in the United States, Spain, and the United Kingdom. IO Biotech is sponsoring the Phase 2 trial and Merck is supplying pembrolizumab.

About IOB-032/PN-E40 Phase 2 Solid Tumor Basket Trial

IOB-032/PN-E40 (NCT05280314) is a multicenter Phase 2 basket trial investigating Cylembio in combination with Merck’s anti-PD-1 therapy, KEYTRUDA (pembrolizumab) as neo-adjuvant/adjuvant treatment of patients with solid tumors at sites in Australia, the United States, France, Germany, Spain, and Denmark. The study completed enrollment in all cohorts: 18 patients with melanoma in cohort A and 16 patients with SCCHN in cohort B, both as single arm cohorts receiving combination of Cylembio with pembrolizumab. In cohort C, 61 melanoma patients were randomized 1:1 to either the combination of Cylembio with pembrolizumab or pembrolizumab alone. In the neo-adjuvant period, for all cohorts, treatment is every 3 weeks (Q3W) for 3 cycles (melanoma) or 2-3 cycles (SCCHN). Patients entering the study will be scheduled for surgery and begin neoadjuvant treatment 4-9 weeks prior. Surgery will be followed by adjuvant treatment with the same regimen for 15 cycles. Cohort C patients with poor pathological response to pembrolizumab alone in the neo-adjuvant phase (>10% residual viable tumor) may cross over to combination treatment post-surgery. The primary endpoint is major pathological response at surgery (≤10% residual viable tumor; central assessment). IO Biotech is sponsoring the Phase 2 trial and Merck is supplying pembrolizumab.

On August 14, 2025 CARsgen Therapeutics Holdings Limited (Stock Code: 2171.HK), a company focused on developing innovative CAR T-cell therapies, reported its 2025 Interim Results (Press release, Carsgen Therapeutics, AUG 14, 2025, View Source [SID1234655321]).

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Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

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Business Highlights

Cash and bank balances were around RMB1,261 million as of June 30, 2025. Cash and cash equivalents and deposits at the end of 2025 are expected to be not less than RMB1,100 million. We expect to have adequate cash into the 2028 excluding subsequent cash inflows.
During H1 2025, certification and regulatory filings for zevor-cel have been completed in more than 20 provinces or cities. CARsgen has received a total of 111 confirmed orders from its commercialization partner Huadong Medicine.
The Center for Drug Evaluation (CDE) of the National Medical Products Administration (NMPA) of China has accepted the New Drug Application (NDA) for satri-cel.
The results of satri-cel confirmatory Phase II trial in China have been published in The Lancet and at the 2025 ASCO (Free ASCO Whitepaper) Annual Meeting.
Multiple allogeneic CAR-T products are in development, covering treatment areas such as hematologic malignancies, solid tumors, and autoimmune diseases.
CARsgen introduced Zhuhai SB Xinchuang to accelerate allogeneic CAR-T development in mainland China.
Dr. Zonghai Li, Founder, Chairman of the Board, Chief Executive Officer, and Chief Scientific Officer of CARsgen Therapeutics, said, "In the first half of 2025, we made significant strides across technology innovation, product development, and commercialization. Zevor-cel sales surged, while satri-cel became the world’s first CAR-T targeting solid tumors to file an NDA. We are also advancing multiple allogeneic CAR-T therapies to enhance clinical outcomes and expand patient access."

Financial Highlights

CARsgen’s revenue was around RMB51 million for the six months ended June 30, 2025, mainly from zevor-cel, which was calculated on the basis of ex-works price, rather than end-of-market prices. Our revenue is recognized upon completion of ex-works delivery of products. Due to the inherent time cycle of CAR-T manufacturing, there is a discrepancy between the number of orders obtained from Huadong Medicine and number of ex-works deliveries. CARsgen’s gross profit was around RMB29 million for the six months ended June 30, 2025. In the commercialization stage, we are demonstrating a strong cost competitive advantage, which is mainly due to self-manufacture for plasmids and vectors with stable output and high yield per batch.

Cash and bank balances were around RMB1,261 million as of June 30, 2025, representing a decrease of around RMB218 million from around RMB1,479 million as of December 31, 2024. The decrease was mainly due to the payment of research and development expenses, administrative expenses and investment of capital expenditure. Cash and cash equivalents and deposits at the end of 2025 are expected to be not less than RMB1,100 million. We expect to have adequate cash into the 2028 excluding subsequent cash inflows.

Zevor-cel Demonstrates Rapid Sales Growth

Zevorcabtagene autoleucel (zevor-cel, R&D code: CT053) is an autologous fully human CAR T-cell product against B-cell maturation antigen (BCMA) approved by the NMPA of China for the treatment of adult patients with relapsed or refractory multiple myeloma (R/R MM) who have progressed after at least 3 prior lines of therapy (including a proteasome inhibitor and an immunomodulatory agent).

CARsgen entered into a collaboration agreement with Huadong Medicine (000963.SZ) for the commercialization of zevor-cel in mainland China. In terms of commercialization, Huadong Medicine has established a dedicated, professional, and comprehensive commercial team to promote the use of zevor-cel and has been utilizing China’s multi-layered insurance system to improve patient accessibility. During the first half of 2025, certification and regulatory filings for zevor-cel have been completed in more than 20 provinces or cities and we have received a total of 111 confirmed orders from Huadong Medicine. We anticipate that growth of sales revenue of zevor-cel will further accelerate with continuous marketing activities and broader insurance coverage.

Satri-cel NDA Accepted for Review in China

Satricabtagene autoleucel (satri-cel, R&D code: CT041) is an autologous humanized CAR T-cell product against Claudin18.2 (CLDN18.2). In June 2025, the CDE of NMPA of China has accepted the New Drug Application (NDA) for satri-cel for the treatment of Claudin18.2-positive advanced gastric/gastroesophageal junction adenocarcinoma (G/GEJA) in patients who have failed at least two prior lines of therapy. Satri-cel is the first and only CAR T-cell product globally for which an NDA submitted for the treatment of solid tumors. Satri-cel was granted Priority Review in May 2025 and Breakthrough Therapy Designation (BTD) in March 2025 by the CDE.

The results of satri-cel confirmatory Phase II trial (NCT04581473) in China have been published in The Lancet and were orally presented at the 2025 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting. Satri-cel demonstrated significant progression-free survival (PFS) improvement and a clinically meaningful overall survival (OS) benefit with a manageable safety profile, compared to standard therapy.

Multiple Allogeneic CAR-T Product Candidates in Development

CARsgen has been advancing differentiated allogeneic CAR T-cell products utilizing the proprietary THANK-uCAR platform. CARsgen has recently developed the THANK-u PlusTM platform as an enhanced version of THANK-uCAR to address the potential impact of NKG2A expression levels on therapeutic efficacy of the allogeneic CAR T-cells.

CT0596 is an allogeneic BCMA-targeted CAR T-cell product utilizing THANK-u Plus platform for the treatment of R/R MM and R/R plasma cell leukemia (PCL). The investigator-initiated trials (IITs) are ongoing in China. Preliminary clinical data for CT0596 were released in May 2025 on CARsgen’s official website. Based on the preliminary safety and efficacy data, CT0596 demonstrated favorable tolerability and encouraging efficacy signals in R/R MM patients across all predefined dose levels, with CAR-T expansion observed.

In addition, there are several allogeneic CAR T-cell products under development:

KJ-C2219: Targeting CD19/CD20, for the treatment of hematologic malignancies and autoimmune diseases. An IIT for relapsed/refractory B-cell non-Hodgkin lymphoma (R/R B-NHL) has been initiated at the end of 2024. A separate IIT for systemic lupus erythematosus (SLE) and systemic sclerosis (SSc) has been initiated in the first half of 2025.
KJ-C2320: Targeting CD38, for the treatment of acute myeloid leukemia (AML). An IIT has been initiated at the end of 2024.
KJ-C2114: For the treatment of solid tumors.
KJ-C2526: Targeting NKG2DL, for the treatment of AML, other malignancies, and senescence.
On February 25, 2025, CARsgen has entered into the agreements (the "Agreements") with an investment fund (the "Investor") managed by Zhuhai Hengqin SB Xinchuang Equity Investment Management Enterprise (Limited Partnership), pursuant to which, among others, the Investor has agreed to subscribe to additional registered capital of UCARsgen Biotech Limited ("UCARsgen") at a cash consideration of RMB80,000,000, representing 8% stake of the enlarged registered capital of UCARsgen. Upon the completion of the capital increase, CARsgen’s share in UCARsgen will be diluted from 100% to 92%.

UCARsgen is a China-based new drug discovery biotechnology company focused on allogeneic CAR T-cell therapies for the treatment of hematologic malignancies. Under the Agreements, UCARsgen has secured the exclusive rights in mainland China for the research, development, manufacture, and commercialization of the following allogeneic CAR T-cell products from CARsgen: the BCMA-targeted allogeneic CAR T-cell therapy for the treatment of multiple myeloma and plasma cell leukemia and the CD19/CD20 dual-targeted allogeneic CAR T-cell therapy for the treatment of B-cell malignancies (excluding indications for the treatment of autoimmune diseases).

On August 14, 2025 Nanjing Leads Biolabs Co., Ltd. ("Leads Biolabs" or the "Company," Stock Code: 9887.HK) reported the successful completion of patient enrollment in the ongoing single-arm, pivotal registrational clinical trial (CTR20213023) for Opamtistomig (LBL-024), a novel PD-L1/4-1BB bispecific antibody (Press release, Nanjing Leads Biolabs, AUG 14, 2025, View Source [SID1234655322]).

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This marks the world’s first registrational clinical trial evaluating an immunotherapy monotherapy for extrapulmonary neuroendocrine carcinoma (EP-NEC). The study, led by Professor Shen Lin from Peking University Cancer Hospital and conducted across multiple medical centers, aims to evaluate the efficacy and safety of Opamtistomig in patients with advanced EP-NEC who failed at least two lines of chemotherapy.

EP-NEC is a highly aggressive, immunologically "cold" tumor. The current first-line standard treatment for advanced EP-NEC remains platinum-based chemotherapy, with an objective response rate (ORR) of 30%-50% and a median overall survival (mOS) of approximately 1 year. For patients with platinum-resistant, no established second-line standard of care exists, representing a pressing unmet need for new therapeutic options.

Dr. Charles Cai, Chief Medical Officer of Leads Biolabs, stated:" As the first PD-L1/4-1BB-targeted bispecific antibody globally to have reached pivotal single-arm registrational stage, Opamtistomig has demonstrated compelling antitumor activity and favorable safety in early trials. Completing enrollment months ahead of schedule accelerates clinical development timelines and reflects both strong confidence of investigators in the trial design and the urgent patient need for effective, safer therapies. We remain committed to advancing rigorous clinical development across multiple indications, including EP-NEC."

Dr. Xiaoqiang Kang, Founder, Chairman and CEO of Leads Biolabs, added, "This achievement was made possible by the exceptional execution of our clinical team, the dedication of investigators, and the trust of patients and their families. Every data point in this study represents hope for patients facing a devastating disease. Guided by our mission-care for life, focus on innovation, and win win cooperation-we will continue to invest in R&D to accelerate Opamtistomig’s global development and deliver transformative therapies worldwide."

About Opamtistomig

Opamtistomig (LBL-024) is a potential first-in-class bispecific antibody simultaneously targeting PD-L1 and the co-stimulatory receptor 4-1BB. It is the first 4-1BB-targeting bispecific antibody globally to reach the single arm pivotal trial stage as a monotherapy and holds promise to become the first approved treatment specifically for extrapulmonary neuroendocrine carcinoma (EP-NEC), a malignancy with significant unmet medical need.

Developed using Leads Biolabs’ proprietary X-Body bispecific platform, Opamtistomig features a 2:2 format with two binding domains each for PD-L1 and 4-1BB, and an optimized affinity ratio. This design allows Opamtistomig to both reverse PD-L1–mediated immune suppression and selectively enhance T cell activation, resulting in potent, synergistic anti-tumor effects.

In Phase I/II clinical trials in China, Opamtistomig has demonstrated promising efficacy and a favorable safety profile in patients with advanced EP-NEC, both as monotherapy and in combination with chemotherapy. The lack of a standard of care in EP-NEC supports the pursuit of accelerated approval through a single-arm pivotal study.

In recognition of its clinical potential, Opamtistomig received Breakthrough Therapy Designation (BTD) from the National Medical Products Administration (NMPA) in China (October 2024), and Orphan Drug Designation (ODD) from the U.S. Food and Drug Administration (FDA) for neuroendocrine carcinoma (November 2024).

Notably, 4-1BB agonism can reactivate exhausted T cells and drive robust proliferation, making it particularly promising for PD-1/PD-L1-resistant or immunologically "cold" tumors. Beyond EP-NEC, Opamtistomig has been approved for clinical trial across multiple cancer types with high unmet need, including small cell lung cancer (SCLC), biliary tract cancer (BTC), ovarian cancer (OC), non-small cell lung cancer (NSCLC), esophageal squamous cell carcinoma (ESCC), hepatocellular carcinoma (HCC), gastric cancer (GC), triple-negative breast cancer (TNBC), and malignant melanoma. Encouraging clinical activity has already been observed in SCLC, BTC, OC, and other cancer types, supporting Opamtistomig’s potential as a broad-spectrum oncology therapy.

About EP-NEC

Neuroendocrine carcinoma (NEC) is a class of poorly differentiated, high-grade neuroendocrine neoplasms (NENs), which originate in the diffuse neuroendocrine cell system and can occur in many different sites. NEC can be categorized into pulmonary NEC and EP-NEC. EP-NEC exhibits similar highly aggressive and metastatic characteristics to small cell lung cancer (SCLC). Most patients are diagnosed at a later stage or already have distant metastases, resulting in rapid disease progression and a poor prognosis.

Currently, the primary first-line treatment for advanced EP-NEC involves platinum-based chemotherapy, achieving an overall response rate (ORR) of approximately 30% to 50%, with a median overall survival (mOS) of only approximately one year. Treatment options for patients with platinum-resistant are limited, and there are no established second-line standard of care for patients who progress after first-line therapy. These underscore the urgency to develop novel therapeutic approaches.

On August 14, 2025 Werewolf Therapeutics, Inc. (the "Company" or "Werewolf") (Nasdaq: HOWL), an innovative biopharmaceutical company pioneering the development of conditionally activated therapeutics engineered to stimulate the body’s immune system for the treatment of cancer and other immune-mediated conditions, reported a business update and announced financial results for the second quarter ended June 30, 2025 (Press release, Werewolf Therapeutics, AUG 14, 2025, View Source [SID1234655254]).

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"We continue to advance the differentiated technology born out of our PREDATOR platform and capitalize on the strong foundation of clinical data from our lead INDUKINETM asset, WTX-124," said Daniel J. Hicklin, Ph.D., President and Chief Executive Officer of Werewolf. "For WTX-124, we remain on track for an interim data readout of our Phase 1/1b clinical trial in the fourth quarter of 2025. The readout is expected to include patients with cutaneous melanoma and renal cell carcinoma, where there exist significant unmet therapeutic needs and which represent large commercial opportunities in oncology. We also anticipate meeting with the FDA later this year to discuss potential registrational pathways. Further, we are excited to introduce WTX-1011, our first INDUCER T-cell engager candidate, targeting STEAP1 for prostate cancer. Utilizing novel anti-CD3 PREDATOR masking technology, our WTX-1011 preclinical data have shown anti-tumor activity while reducing peripheral activity to prevent cytokine release syndrome. Given the broad potential of our approach with INDUCER molecules, we plan to nominate an additional candidate before year-end."

Recent Highlights and Upcoming Milestones

Clinical-Stage INDUKINE Molecules:
•WTX-124: a systemically delivered, conditionally activated Interleukin-2 (IL-2) INDUKINE molecule being developed as monotherapy and in combination with pembrolizumab in multiple solid tumor types.

◦All expansion arms are actively enrolling patients in the ongoing Phase 1/1b clinical trial at a recommended dose of 18 mg administered intravenously every two weeks (IV Q2W).

◦During the fourth quarter of 2025, Werewolf plans to release interim data from the monotherapy and combination expansion arms, including tolerability, response rate, and durability for patients with cutaneous melanoma and renal cell carcinoma.

◦Werewolf expects to engage with regulatory authorities in the second half of 2025 to discuss potential registrational pathways for WTX-124 in advanced or metastatic cutaneous melanoma.
•WTX-330: a systemically delivered, conditionally activated Interleukin-12 (IL-12) INDUKINE molecule being developed in advanced or metastatic solid tumors.
◦Actively enrolling in a Phase 1b/2 clinical trial (WTX-330×2102) in locally advanced or metastatic solid tumors. Anticipate determination of dosing regimen by the end of 2025.

Preclinical-Stage INDUCER Molecules:
•WTX-1011: a potential first-in-class conditionally activated anti-STEAP1 T-cell engager to provide an improved therapeutic index.
◦STEAP1 is a promising prostate cancer target with limited expression in normal tissues, but notable toxicities are associated with existing anti-STEAP1 T-cell engager therapy.
◦Preclinical data demonstrated that PREDATOR masking technology successfully silenced peripheral activity and prevented cytokine release.
▪WTX-1011 has beneficial exposure in cynomolgus monkeys with a half-life close to 100 hours.
▪WTX-1011 is stable in the periphery, with less than 0.7% of active INDUCER molecule detected.
▪Effective masking of WTX-1011 is confirmed by low levels of cytokine production compared to a 10-fold lower dose of unmasked STEAP1 INDUCER molecule.
•Utilizing a novel and highly effective anti-CD3 masking strategy, Werewolf expects to nominate an additional candidate in the second half of 2025.
Preclinical-Stage INDUKINE Portfolio:
•Werewolf’s previously announced development candidates WTX-712, its Interleukin-21 (IL-21) INDUKINE molecule, and WTX-518, its binding protein resistant Interleukin-18 (IL-18) INDUKINE molecule, each for the treatment of cancer, and WTX-921, a first-of-its-kind Interleukin-10 (IL-10) INDUKINE molecule for the treatment of inflammatory bowel disease (IBD) and potentially other inflammatory diseases, are available for partnering.
Financial Results for the Second Quarter of 2025:
•Cash position: As of June 30, 2025, cash and cash equivalents were $77.6 million, compared to $92.0 million as of March 31, 2025. The Company believes its existing cash and cash equivalents as of June 30, 2025, will be sufficient to fund operational expenses and capital expenditure requirements into the fourth quarter of 2026.
•Research and development expenses: Research and development expenses were $13.1 million for the second quarter of 2025, compared to $15.3 million for the same period in 2024.
•General and administrative expenses: General and administrative expenses were $4.4 million for the second quarter of 2025, compared to $4.8 million for the same period in 2024.
•Net loss: Net loss was $18.0 million for the second quarter of 2025, compared to $17.2 million for the same period in 2024.